Article

Gene Therapy Restores the Transcriptional Program of Hematopoietic Stem Cells in Fanconi Anemia

2021-07-22

Abstract excerpt

<h4>ABSTRACT</h4> Fanconi anemia (FA) is an inherited disease associated with marked hematopoietic stem and progenitor cell (HSPC) defects. Ongoing clinical trials have shown that lentiviral-mediated gene therapy can ameliorate bone marrow failure (BMF) in non-conditioned FA patients thanks to the proliferative advantage of corrected FA HSPCs. Here we investigated whether gene therapy can revert affected molecular...

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Literature Corpus work: 845710c6-6756-5271-97b0-bb75263b1e92
DOI: 10.1101/2021.07.20.21260460

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